Phuturdex › Diseases › Leber hereditary optic neuropathy
Drugs in development · updated 30 October 2026
Leber hereditary optic neuropathy: drugs in development
As of 30 October 2026 there are 6 active clinical trials (recruiting or ongoing, phases I–III) on 4 drugs for leber hereditary optic neuropathy. 4 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| NR082 | II–III | 2 | NCT04912843 |
| Sepofarsen | II–III | 2 | NCT03913143 |
| GS010 | II | 1 | NCT07303296 |
| NicotinamideNicotinomide | I | 1 | NCT07258667 |
Sorted by most advanced phase and number of active trials.
All 4 drugs, with the details
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Open in Phuturdex →Broader categories
- Metabolic disease 30 drugs
- Nutritional and metabolic diseases 5 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Cranial nerve disease 4 drugs
- Nervous system heredodegenerative disease 3 drugs
- Nervous System Diseases 39 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D029242 “Optic Atrophy, Hereditary, Leber”. Prevalence: Orphanet.